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Congenital, familial and genetic disorders – page 41

  • 796 clinical trials
  • 308 recruiting
  • 374 conditions
  • 27 countries

Conditions in this area

374 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Spinal muscular atrophy17Hypertrophic cardiomyopathy16Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Sickle cell anaemia13Congenital cystic kidney disease12Huntington's disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Fabry's disease10Hereditary angioedema with C1 esterase inhibitor deficiency10+350

Clinical trials in Congenital, familial and genetic disorders

796 trials across Europe, recruiting first

Type of trial
Recruitment status
Quick filters

796 clinical trials

Factor IX deficiency

Not recruiting

Title: Study of gene therapy Etranacogene Dezaparvovec (Hemgenix) in adult patients with moderate to severe Hemophilia B

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Etranacogene Dezaparvovec
  • Sponsor:CSL Behring LLC

CDKL5 deficiency disorder

Not recruiting

Study on the Effectiveness and Safety of Fenfluramine Hydrochloride for Patients with CDKL5 Deficiency Disorder

Registered drug
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years
  • Substances:Fenfluramine Hydrochloride
  • Sponsor:Zogenix International Limited

Facioscapulohumeral muscular dystrophy

Not recruiting

Study on the Effectiveness and Safety of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)

Investigational
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Losmapimod
  • Sponsor:Fulcrum Therapeutics Inc.

Duchenne muscular dystrophy+1

Not recruiting

Study on the Safety and Effects of Delandistrogene Moxeparvovec in Children Under 4 with Duchenne Muscular Dystrophy

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:Delandistrogene Moxeparvovec
  • Sponsor:F. Hoffmann-La Roche AG

Activated PI3 kinase delta syndrome

Not recruiting

Study on the Effects of Leniolisib in Children Aged 1 to 6 with Activated PI3K Delta Syndrome (APDS)

InvestigationalNo placebo
Countries:PortugalPortugal
  • Participants:0–17 years
  • Substances:Leniolisib Phosphate
  • Sponsor:Pharming Technologies B.V.

Glycogen storage disease type II

Not recruiting

Long-Term Safety Study of Avalglucosidase Alfa for Patients with Pompe Disease in France

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Avalglucosidase Alfa
  • Sponsor:Sanofi Winthrop Industrie

Myotonic dystrophy

Not recruiting

Study on AOC 1001 for Treating Myotonic Dystrophy Type 1 in Patients

Investigational
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years
  • Substances:Humanised Igg1 Monoclonal Antibody Against Tfr1 Conjugated To Double Stranded Sirna Oligonucleotide Against Dmpk Via A Non-Cleavable Linker
  • Sponsor:Avidity Biosciences Inc.

Spinal muscular atrophy

Not recruiting

Long-term Safety Study of Onasemnogene Abeparvovec for Patients with Spinal Muscular Atrophy

Registered drugNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years
  • Substances:Onasemnogene Abeparvovec
  • Sponsor:Novartis Pharma AG

Amyotrophic lateral sclerosis+2

Not recruiting

Study of Tofersen for Adults with Presymptomatic Amyotrophic Lateral Sclerosis (ALS) Due to SOD1 Gene Mutation

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Tofersen
  • Sponsor:Biogen Idec Research Limited

Asplenia

Not recruiting

Study on the Safety and Immune Response of Meningococcal B Vaccines in Adults with Asplenia: Bexsero, Trumenba, and a Drug Combination

Registered drugNo placebo
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:Neisseria Meningitidis Group B Fhbp Protein Subfamily A
  • Sponsor:Assistance Publique Hopitaux De Paris

Colorectal cancer metastatic+2

Not recruiting

Study on the Safety and Efficacy of CR6086 and Balstilimab for Patients with Pretreated Metastatic Colorectal and Other GI Cancers

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:18–64 years · 65+ years
  • Substances:Balstilimab
  • Sponsor:Rottapharm Biotech S.r.l.

Pyruvate kinase deficiency anaemia

Not recruiting

Study on the Effectiveness and Safety of Mitapivat for Children with Pyruvate Kinase Deficiency Receiving Regular Blood Transfusions

Investigational
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Mitapivat
  • Sponsor:Agios Pharmaceuticals Inc.

Substances studied in this area

The therapies tested most often in Congenital, familial and genetic disorders trials.

Sirolimus21 clinical trialsSodium Chloride21 clinical trialsPrednisolone12 clinical trialsPlerixafor11 clinical trialsBusulfan10 clinical trialsIvacaftor9 clinical trialsMitapivat9 clinical trialsParacetamol8 clinical trialsTezacaftor8 clinical trials
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